20 completed, 12 active not recruiting, 12 recruiting, 6 terminated. 28 Phase 2, 12 Phase 1, 5 Not applicable, 4 Phase 3, 1 Early Phase 1.
Follow Neurofibroma, Plexiform: new trials by email Literature Review of Neurofibroma, Plexiform trials Research Report on Neurofibroma, Plexiform
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Study of Cabozantinib With Selumetinib for Plexiform Neurofibromas Cabozantinib Oral Tablet, Selumetinib Oral Capsule - Neurofibromatosis 1, Plexiform Neurofibroma | Phase 1 | Recruiting | Girish Dhall, MD | 30 | 2026-06-09 | 2026-10-02 |
| Evaluation of TQ-B3234 Capsules in Patients With Symptomatic, Non-Surgical Type 1 Neurofibromatosis-Associated Plexiform Neurofibromas TQ-B3234 capsules, TQ-B3234 placebo - Plexiform Neurofibroma | Phase 3 | Recruiting | Chia Tai Tianqing Pharmaceutical Group Co., Ltd. | 177 | 2026-03-31 | 2026-05-27 |
| A Study to Evaluate the Feasibility of a Physiologic Biomarker to Assess Pain and Other Sensory Problems Using Pupillometry in Participants With Neurofibromatosis Type 1 (NF1) AlgometRx Nociometer(R) - Neurofibromatosis Type 1 | Not applicable | Recruiting | National Cancer Institute (NCI) | 70 | 2026-02-18 | 2026-10-16 |
| Selumetinib for the Prevention of Plexiform Neurofibroma Growth in NF Type 1 Selumetinib - Neurofibromatosis 1, Plexiform Neurofibroma | Phase 2 | Recruiting | University of Alabama at Birmingham | 200 | 2025-08-27 | 2026-05-29 |
| Follow-up Study to Evaluate the Safety and Efficacy of FCN-159 in Pediatric Participants With Neurofibromatosis Type 1 Luvometinib Tablets - Neurofibromatosis 1, Plexiform Neurofibroma | Phase 1 / Phase 2 | Not yet recruiting | Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. | 65 | 2025-06-12 | 2025-06-17 |
| PAS-004 in Adults Who Have Neurofibromatosis Type 1 With Plexiform Neurofibromas PAS-004 Tablets, PAS-004 Capsule - NF1 Mutation, Neurofibroma Plexiform | Phase 1 | Recruiting | Pasithea Therapeutics Corp. | 56 | 2025-05-30 | 2026-10-28 |
| A Phase 2, Open-Label Study to Evaluate the Safety and Effects of HLX-1502 in Patients With Neurofibromatosis Type 1 HLX-1502 - Neurofibromatosis Type 1 | Phase 2 | Recruiting | Healx Limited | 25 | 2025-02-10 | 2026-07-29 |
| Low-dose Selumetinib for the Treatment of Plexiform Neurofibromas in Chinese Children Selumetinib - Plexiform Neurofibromas (PN) | Phase 2 | Not yet recruiting | West China Hospital | 50 | 2025-01-01 | 2025-01-08 |
| Surveillance for Malignant Transformation of Neurofibromatosis Type 1 (NF1) Related Peripheral Nerve Sheath Tumors (PNST) Neurofibromatosis 1, Nerve Sheath Neoplasms | Recruiting | National Cancer Institute (NCI) | 225 | 2024-10-09 | 2026-11-03 | |
| Neurofibromatosis Type 1 Tumor Early Detection Study Liquid biopsy for MPNST development - Neurofibromatosis Type 1, Neurofibromatosis 1 | Enrolling by invitation | David Miller | 1000 | 2024-08-07 | 2026-08-31 | |
| Real-World Treatment Study of Koselugo (Selumetinib) Neurofibromatosis 1, Neurofibroma, Plexiform | Recruiting | AstraZeneca | 200 | 2024-06-15 | 2026-08-25 | |
| Observational Study to Evaluate the Effect and Safety of Selumetinib in Pediatric Patients With NF1-PNs Neurofibroma, Plexiform | Active not recruiting | AstraZeneca | 409 | 2023-12-20 | 2026-09-23 | |
| A Study of Avutometinib for People With Solid Tumor Cancers Avutometinib - Refractory Cancer, CNS Tumors | Phase 1 | Completed | Memorial Sloan Kettering Cancer Center | 3 | 2023-10-20 | 2026-11-04 |
| Selumetinib in Chinese Paediatric With Post-operative NF1-PNs, PhaseⅡ, Double-Blinded, Placebo-Controlled Study Selumetinib, Placebo - Neurofibromatosis Type 1, Plexiform Neurofibromas | Phase 2 | Withdrawn | AstraZeneca | 2023-08-31 | 2023-08-14 | |
| FCN-159 in Adult Patients With Symptomatic, Inoperable Neurofibromatosis Type 1-Related Plexiform Neurofibromas Test group (Group A): FCN-159 8 mg, orally, once daily;, Control group (Group B): Placebo, orally, once daily; - Neurofibromatosis 1, Plexiform Neurofibroma | Phase 3 | Active not recruiting | Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. | 167 | 2023-06-20 | 2025-03-06 |
| Developing Biomarkers of Plexiform Tumor Burden in Patients With Neurofibromatosis-Type 1 Neurofibromatosis 1, NF1 | Enrolling by invitation | Ann & Robert H Lurie Children's Hospital of Chicago | 200 | 2022-03-04 | 2026-09-09 | |
| Evaluation of Percutaneous Cryotherapy in the Treatment of Plexiform Neurofibromas and Unresectable Neurofibromas in Neurofibromatosis Type 1 Cryotherapy - Neurofibroma, Neurofibroma, Plexiform | Not applicable | Recruiting | Centre Leon Berard | 30 | 2022-02-14 | 2024-02-09 |
| Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas Selumetinib, Placebo - Neurofibromatosis 1, Plexiform Neurofibroma (PN) | Phase 3 | Active not recruiting | AstraZeneca | 145 | 2021-11-19 | 2026-03-23 |
| HL-085 in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas HL-085 - Neurofibromatosis 1, Plexiform Neurofibromas | Phase 2 | Recruiting | Shanghai Kechow Pharma, Inc. | 70 | 2021-10-18 | 2023-05-31 |
| Study to Evaluate the Safety, Tolerability, PK Characteristics and Anti-tumor Activity of FCN-159 in Adult and Pediatric Participants With Neurofibromatosis Type 1 FCN-159 - Neurofibromatosis 1, Plexiform Neurofibroma | Phase 1 / Phase 2 | Active not recruiting | Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. | 160 | 2021-03-26 | 2024-11-12 |
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Neurofibromatosis 1 (43) · Neurofibromatoses (10) · Neurofibroma (9) · Neoplasms (4) · Brain Neoplasms (3) · Neuroblastoma (3) · Neurofibrosarcoma (3) · Optic Nerve Glioma (3) · Precancerous Conditions (3) · Glioma (2) · Histiocytosis, Langerhans-Cell (2) · Nerve Sheath Neoplasms (2) · Pain (2) · Sarcoma (2)
Neoplasms by Histologic Type (62) · Nervous System Diseases (62) · Nervous System Neoplasms (62) · Neuromuscular Diseases (62) · Neoplasms, Nerve Tissue (61) · Peripheral Nervous System Diseases (61) · Peripheral Nervous System Neoplasms (61) · Nerve Sheath Neoplasms (60) · Neoplasms (58) · Neurofibroma (53) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (49) · Genetic Diseases, Inborn (49) · Neoplastic Syndromes, Hereditary (49) · Neurocutaneous Syndromes (48) · Neurodegenerative Diseases (48)
AZD 6244 (11) · trametinib (4) · pirfenidone (3) · Imatinib Mesylate (2) · cabozantinib (2) · dabrafenib (2) · mirdametinib (2) · Acceptance and Commitment Therapy (1) · Control Groups (1) · Cryotherapy (1) · Everolimus (1) · Fluorodeoxyglucose F18 (1) · Gadolinium DTPA (1) · Liquid Biopsy (1) · Methotrexate (1)
National Cancer Institute (NCI) (18) · AstraZeneca (6) · Indiana University (3) · Novartis Pharmaceuticals (3) · Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. (3) · University of Alabama at Birmingham (3) · Children's Hospital of Philadelphia (2) · SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany (2) · St. Justine's Hospital (2) · Alexion Pharmaceuticals, Inc. (1) · Ann & Robert H Lurie Children's Hospital of Chicago (1) · Assistance Publique - Hôpitaux de Paris (1) · Centre Leon Berard (1) · Chia Tai Tianqing Pharmaceutical Group Co., Ltd. (1) · Children's National Research Institute (1)
United States (43) · China (10) · Canada (5) · France (5) · Australia (4) · Germany (4) · United Kingdom (4) · Spain (3) · Brazil (2) · Israel (2) · Italy (2) · Japan (2) · Russia (2) · South Korea (2) · Sweden (2)
Brigitte C Widemann, M.D. (9) · Chie-Schin Shih, MD (2) · Miriam Bornhorst, MD (2) · Pamela L Wolters, Ph.D. (2) · Staci M Peron, Ph.D. (2) · Alice P. Chen, MD (1) · Brigitte C. Widemann, MD (1) · Brigitte Widemann, M.D. (1) · Bruce Korf, MD, PhD (1) · Carlos Prada, MD (1) · Darren Hargrave, MB Bch (1) · David T Miller, MD, PhD (1) · Dusica Babovic-Vuksanovic (1)
PMID 35007270, PMID 24321536, PMID 24500418, PMID 24753394, PMID 27510726, PMID 28029918, PMID 30339775, PMID 31881853, PMID 32152628, PMID 32187457, PMID 32562630, PMID 33442015, PMID 33536190, PMID 34331515, PMID 35467749, PMID 36375115, PMID 36884302, PMID 37115514, PMID 37400844, PMID 38869947, PMID 39116355, PMID 39418437, PMID 39514826, PMID 39762421, PMID 40473450, PMID 40709016, PMID 41139346, PMID 42394972, PMID 42540436
2018 (1), 2023 (1), N01CM17104 (1), N01CM62205 (1), P30CA015083 (1), R33NS122094 (1), R61NS122094 (1)